JP2010525084A - 薬理シャペロンを用いたリソソーム蓄積症治療のための投薬計画 - Google Patents

薬理シャペロンを用いたリソソーム蓄積症治療のための投薬計画 Download PDF

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JP2010525084A
JP2010525084A JP2010506557A JP2010506557A JP2010525084A JP 2010525084 A JP2010525084 A JP 2010525084A JP 2010506557 A JP2010506557 A JP 2010506557A JP 2010506557 A JP2010506557 A JP 2010506557A JP 2010525084 A JP2010525084 A JP 2010525084A
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デイビッド・ロックハート
ブランドン・ウストマン
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アミカス セラピューティックス インコーポレイテッド
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    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/33Heterocyclic compounds
    • A61K31/395Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
    • A61K31/435Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with one nitrogen as the only ring hetero atom
    • A61K31/44Non condensed pyridines; Hydrogenated derivatives thereof
    • A61K31/445Non condensed piperidines, e.g. piperocaine
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/33Heterocyclic compounds
    • A61K31/395Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
    • A61K31/435Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with one nitrogen as the only ring hetero atom
    • A61K31/44Non condensed pyridines; Hydrogenated derivatives thereof
    • A61K31/445Non condensed piperidines, e.g. piperocaine
    • A61K31/45Non condensed piperidines, e.g. piperocaine having oxo groups directly attached to the heterocyclic ring, e.g. cycloheximide
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • A61P25/14Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
    • A61P25/16Anti-Parkinson drugs
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • A61P25/28Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00Drugs for disorders of the metabolism
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P7/00Drugs for disorders of the blood or the extracellular fluid
    • A61P7/06Antianaemics
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00Drugs for disorders of the cardiovascular system

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  • Health & Medical Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Animal Behavior & Ethology (AREA)
  • General Health & Medical Sciences (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Organic Chemistry (AREA)
  • General Chemical & Material Sciences (AREA)
  • Epidemiology (AREA)
  • Neurosurgery (AREA)
  • Biomedical Technology (AREA)
  • Neurology (AREA)
  • Diabetes (AREA)
  • Hematology (AREA)
  • Obesity (AREA)
  • Hospice & Palliative Care (AREA)
  • Heart & Thoracic Surgery (AREA)
  • Cardiology (AREA)
  • Psychiatry (AREA)
  • Psychology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Acyclic And Carbocyclic Compounds In Medicinal Compositions (AREA)
  • Hydrogenated Pyridines (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
JP2010506557A 2007-04-26 2008-04-28 薬理シャペロンを用いたリソソーム蓄積症治療のための投薬計画 Pending JP2010525084A (ja)

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US91428807P 2007-04-26 2007-04-26
US1474407P 2007-12-18 2007-12-18
US2810508P 2008-02-12 2008-02-12
PCT/US2008/061764 WO2008134628A2 (en) 2007-04-26 2008-04-28 Dosing regimens for the treatment of lysosomal storage diseases using pharmacological chaperones

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JP2010525084A true JP2010525084A (ja) 2010-07-22
JP2010525084A5 JP2010525084A5 (enExample) 2011-05-19

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US (2) US9056101B2 (enExample)
EP (1) EP2150254A4 (enExample)
JP (1) JP2010525084A (enExample)
AU (1) AU2008245578A1 (enExample)
CA (1) CA2685332A1 (enExample)
IL (1) IL201733A0 (enExample)
MX (1) MX2009011473A (enExample)
WO (1) WO2008134628A2 (enExample)

Cited By (10)

* Cited by examiner, † Cited by third party
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JP2015519329A (ja) * 2012-05-03 2015-07-09 アミカス セラピューティックス インコーポレイテッド ポンペ病の処置のための投与計画
JP2017511372A (ja) * 2014-04-14 2017-04-20 アミカス セラピューティックス インコーポレイテッド 脳アミロイドーシスを処置及び/又は予防する投与計画
JP2017161528A (ja) * 2011-06-06 2017-09-14 セントジーン アーゲー ゴーシェ病の診断のための方法
JP2018528953A (ja) * 2015-08-31 2018-10-04 アミカス セラピューティックス インコーポレイテッド リソソーム障害及び中枢神経系の変性障害の治療及び予防のための(3r,4r,5s)−5−(ジフルオロメチル)ピペリジン−3,4−ジオールを含むレジメン
US10208299B2 (en) 2014-09-30 2019-02-19 Amicus Therapeutics, Inc. Highly potent acid alpha-glucosidase with enhanced carbohydrates
US10227577B2 (en) 2016-03-30 2019-03-12 Amicus Therapeutics, Inc. Method for selection of high M6P recombinant proteins
US10512676B2 (en) 2016-03-30 2019-12-24 Amicus Therapeutics, Inc. Formulations comprising recombinant acid alpha-glucosidase
JP2020507562A (ja) * 2017-05-30 2020-03-12 アミカス セラピューティックス インコーポレイテッド 腎機能障害を有するファブリー患者の治療方法
US10857212B2 (en) 2015-12-30 2020-12-08 Amicus Therapeutics, Inc. Augmented acid alpha-glucosidase for the treatment of Pompe disease
US12246062B2 (en) 2017-05-15 2025-03-11 Amicus Therapeutics, Inc. Recombinant human acid alpha-glucosidase

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JP2009537149A (ja) 2006-05-16 2009-10-29 アミークス セラピューティックス インコーポレイテッド ファブリー病に対する治療の選択を診断し、評価するためのアッセイ
US9999618B2 (en) 2007-04-26 2018-06-19 Amicus Therapeutics, Inc. Dosing regimens for the treatment of lysosomal storage diseases using pharmacological chaperones
WO2009102895A2 (en) 2008-02-12 2009-08-20 Amicus Therapeutics, Inc. Method to predict response to pharmacological chaperone treatment of diseases
EP2484371B1 (en) 2008-06-26 2014-12-17 Orphazyme APS Use of Hsp70 as a regulator of enzymatic activity
WO2010048532A1 (en) 2008-10-24 2010-04-29 Amicus Therapeutics, Inc. Multiple compartment dosing model
WO2010056746A1 (en) * 2008-11-11 2010-05-20 Amicus Therapeutics, Inc. Therapy regimens, dosing regimens and stable medicaments for the treatment of pompe disease
BRPI1010517A2 (pt) 2009-04-09 2017-01-31 Amicus Therapeutics Inc métodos para prevenção e/ou tratamento de doenças de armazenamento lisossomal
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PT2995306T (pt) 2009-10-19 2019-03-29 Amicus Therapeutics Inc Novas composições para prevenção e/ou tratamento de disfunções de armazenamento lisossomal
JP6061677B2 (ja) * 2009-10-19 2017-01-18 アミカス セラピューティックス インコーポレイテッド 薬理シャペロンを用いてガングリオシドの活性を増大させて、アルツハイマー病を治療するための方法
DK2490532T3 (en) 2009-10-19 2017-02-27 Amicus Therapeutics Inc Hitherto UNKNOWN COMPOSITIONS FOR PREVENTION AND / OR TREATMENT OF DEGENERATIVE DISORDERS IN THE CENTRAL Nervous System
WO2011051346A1 (en) * 2009-10-27 2011-05-05 Erytech Pharma Composition to induce specific immune tolerance
AR079057A1 (es) * 2009-11-17 2011-12-21 Baylor Res Inst Triaosilceramida urinaria (gb3) como marcador de enfermedad cardiaca
SG10201407881WA (en) 2009-11-27 2015-01-29 Genzyme Corp An amorphous and a crystalline form of genz 112638 hemitartrate as inhibitor of glucosylceramide synthase
HUE030932T2 (en) 2010-11-08 2017-06-28 Amicus Therapeutics Inc Increased stability and increased catalytic activity, altered, recombinant, β-glucocerebrosidase proteins
RU2013125923A (ru) 2010-11-30 2015-01-10 Орфазиме Апс СПОСОБЫ УВЕЛИЧЕНИЯ ВНУТРИКЛЕТОЧНОЙ АКТИВНОСТИ Hsp70
US20140219986A1 (en) * 2011-03-11 2014-08-07 Amicus Therapeutics ,Inc. Dosing regimens for the treatment of fabry disease
KR20230066482A (ko) 2012-03-07 2023-05-15 아미쿠스 세라퓨틱스, 인코포레이티드 폼페병의 치료를 위한 고농도 알파-글루코시다제 조성물
CA2868459A1 (en) 2012-03-27 2013-10-03 Amicus Therapeutics, Inc. Polymorphs of (3r,4r,5s)-5-(difluoromethyl)piperidine-3,4-diol fumarate and uses thereof
HK1209625A1 (en) 2012-07-17 2016-04-08 Amicus Therapeutics, Inc. Alpha-galactosidase a and 1-deoxygalactonojirimycin co-formulation
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US10709700B2 (en) 2014-09-15 2020-07-14 Orphazyme A/S Arimoclomol formulation
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WO2017041051A1 (en) 2015-09-04 2017-03-09 Sqz Biotechnologies Company Intracellular delivery of biomolecules to cells comprising a cell wall
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EP3442530A1 (en) 2016-04-13 2019-02-20 Orphazyme A/S Heat shock proteins and cholesterol homeostasis
SI3448382T1 (sl) 2016-04-29 2021-03-31 Orphazyme A/S, c/o COBIS A/S Arimoclomol za zdravljenje motenj, povezanih z glukocerebrozidazo
RU2770492C2 (ru) 2016-05-03 2022-04-18 ЭсКьюЗед БАЙОТЕКНОЛОДЖИЗ КОМПАНИ Внутриклеточная доставка биологических молекул для индуцирования толерантности
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PT4324522T (pt) * 2017-05-30 2026-03-13 Amicus Therapeutics Inc Métodos de tratamento de pacientes com fabry tendo insuficiência renal
EP3749307B1 (en) 2018-02-06 2025-12-10 Amicus Therapeutics, Inc. Use of migalastat for treating fabry disease in pregnant patients
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EP3894864A2 (en) * 2018-12-10 2021-10-20 Denali Therapeutics Inc. Lysosomal storage disorder biomarkers and methods of use thereof
BR112021024886A2 (pt) * 2019-06-11 2022-01-25 Amicus Therapeutics Inc Métodos de tratar a doença de fabry em pacientes com insuficiência renal
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Cited By (29)

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JP2017161528A (ja) * 2011-06-06 2017-09-14 セントジーン アーゲー ゴーシェ病の診断のための方法
JP2020045346A (ja) * 2012-05-03 2020-03-26 アミカス セラピューティックス インコーポレイテッド ポンペ病の処置のための投与計画
JP2018109003A (ja) * 2012-05-03 2018-07-12 アミカス セラピューティックス インコーポレイテッド ポンペ病の処置のための投与計画
JP7046241B2 (ja) 2012-05-03 2022-04-01 アミカス セラピューティックス インコーポレイテッド ポンペ病の処置のための投与計画
JP2015519329A (ja) * 2012-05-03 2015-07-09 アミカス セラピューティックス インコーポレイテッド ポンペ病の処置のための投与計画
JP2021088561A (ja) * 2012-05-03 2021-06-10 アミカス セラピューティックス インコーポレイテッド ポンペ病の処置のための投与計画
JP2017511372A (ja) * 2014-04-14 2017-04-20 アミカス セラピューティックス インコーポレイテッド 脳アミロイドーシスを処置及び/又は予防する投与計画
US11591583B2 (en) 2014-09-30 2023-02-28 Amicus Therapeutics, Inc. Highly potent acid alpha-glucosidase with enhanced carbohydrates
US11753632B2 (en) 2014-09-30 2023-09-12 Amicus Therapeutics, Inc. Highly potent acid alpha-glucosidase with enhanced carbohydrates
US10208299B2 (en) 2014-09-30 2019-02-19 Amicus Therapeutics, Inc. Highly potent acid alpha-glucosidase with enhanced carbohydrates
US10961522B2 (en) 2014-09-30 2021-03-30 Amicus Therapeutics, Inc. Highly potent acid alpha-glucosidase with enhanced carbohydrates
JP2018528953A (ja) * 2015-08-31 2018-10-04 アミカス セラピューティックス インコーポレイテッド リソソーム障害及び中枢神経系の変性障害の治療及び予防のための(3r,4r,5s)−5−(ジフルオロメチル)ピペリジン−3,4−ジオールを含むレジメン
US12414985B2 (en) 2015-12-30 2025-09-16 Amicus Therapeutics, Inc. Augmented acid alpha-glucosidase for the treatment of Pompe disease
US10857212B2 (en) 2015-12-30 2020-12-08 Amicus Therapeutics, Inc. Augmented acid alpha-glucosidase for the treatment of Pompe disease
US11278601B2 (en) 2015-12-30 2022-03-22 Amicus Therapeutics, Inc. Augmented acid alpha-glucosidase for the treatment of Pompe disease
US11491211B2 (en) 2016-03-30 2022-11-08 Amicus Therapeutics, Inc. Formulations comprising recombinant acid alpha-glucosidase
US10512676B2 (en) 2016-03-30 2019-12-24 Amicus Therapeutics, Inc. Formulations comprising recombinant acid alpha-glucosidase
US12622952B2 (en) 2016-03-30 2026-05-12 Amicus Therapeutics, Inc. Formulations comprising recombinant acid α-glucosidase
US10227577B2 (en) 2016-03-30 2019-03-12 Amicus Therapeutics, Inc. Method for selection of high M6P recombinant proteins
US11441138B2 (en) 2016-03-30 2022-09-13 Amicus Therapeutics, Inc. Method for selection of high M6P recombinant proteins
US12246062B2 (en) 2017-05-15 2025-03-11 Amicus Therapeutics, Inc. Recombinant human acid alpha-glucosidase
KR102427610B1 (ko) 2017-05-30 2022-07-29 아미쿠스 세라퓨틱스, 인코포레이티드 신장 손상을 갖는 파브리 환자를 치료하는 방법
JP2020507562A (ja) * 2017-05-30 2020-03-12 アミカス セラピューティックス インコーポレイテッド 腎機能障害を有するファブリー患者の治療方法
JP2023052005A (ja) * 2017-05-30 2023-04-11 アミカス セラピューティックス インコーポレイテッド 腎機能障害を有するファブリー患者の治療方法
JP2023100658A (ja) * 2017-05-30 2023-07-19 アミカス セラピューティックス インコーポレイテッド 腎機能障害を有するファブリー患者の治療方法
JP2020073500A (ja) * 2017-05-30 2020-05-14 アミカス セラピューティックス インコーポレイテッド 腎機能障害を有するファブリー患者の治療方法
KR20210066032A (ko) * 2017-05-30 2021-06-04 아미쿠스 세라퓨틱스, 인코포레이티드 신장 손상을 갖는 파브리 환자를 치료하는 방법
JP2022071094A (ja) * 2017-05-30 2022-05-13 アミカス セラピューティックス インコーポレイテッド 腎機能障害を有するファブリー患者の治療方法
JP2020203886A (ja) * 2017-05-30 2020-12-24 アミカス セラピューティックス インコーポレイテッド 腎機能障害を有するファブリー患者の治療方法

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IL201733A0 (en) 2010-06-16
WO2008134628A2 (en) 2008-11-06
US9056101B2 (en) 2015-06-16
CA2685332A1 (en) 2008-11-06
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